UCL and GSK join forces to develop combined small molecule-antibody treatment for rare disease

Published on 11 March 2009

A collaboration to develop a world first drug-antibody dual treatment for the rare and often fatal condition amyloidosis has been formed between the University College London spinout company Pentraxin Therapeutics Ltd and GlaxoSmithKline (GSK).

Amyloidosis is a disease caused by build up of abnormal proteins (amyloid) in body tissues, leading to organ failure. The heart, kidneys, liver and almost any other organ can be affected. Around 500 new cases are diagnosed each year in the UK. Despite the best available therapy, the prognosis for patients with amyloidosis is poor and new treatments are urgently needed.

“We initially developed the small molecule drug, CPHPC, and while we had promising early results, they were not enough to benefit patients with advanced disease. Something more dramatic is needed,” explained Professor Mark Pepys FRS, the head of Pentraxin and the UCL Centre for Amyloidosis and Acute Phase Proteins which includes the UK National Amyloidosis Centre. “We then combined CPHPC treatment with an antibody that seeks out the amyloid deposits in the organs in mice. This combination triggered a rapid clearance of the deposits.”

With this new agreement, the research teams from UCL and GSK will work together to convert the mouse antibody into one that can be used in humans in combination with CPHPC. The aim is to find out if the benefits seen in the animal model can be replicated in patients with amyloidosis.

The collaboration brings together UCL’s clinical and science expertise and the development expertise of GSK’s Academic Discovery Performance and Biopharm Units.

“We are delighted to enter into this alliance,” said Mike Owen, Senior Vice President, Biopharmaceutical Research, GSK. “Our biopharmaceutical and clinical development capabilities and Prof Pepys’s team’s knowledge of the disease provide a synergistic collaboration that will greatly enhance our chances of success.”

Under the terms of the agreement, Pentraxin will receive undisclosed early stage success-based milestones plus drug development milestones and royalties.

About Pentraxin Therapeutics

Pentraxin Therapeutics Ltd is a company spun out from University College London (UCL) by UCL Business PLC (UCLB) to hold and develop the intellectual property of Professor Mark Pepys and his colleagues in the UCL Centre for Amyloidosis and Acute Phase Proteins. This clinical and basic science research centre houses the UK NHS National Amyloidosis Centre and it leads the world in both research and clinical management of amyloidosis (

About GlaxoSmithKline

GlaxoSmithKline – one of the world’s leading research-based pharmaceutical and healthcare companies – is committed to improving the quality of human life by enabling people to do more, feel better and live longer. For company information, visit GlaxoSmithKline at


To view a video about systemic amyloidosis please visit

Further Information

Professor Mark Pepys, UCL/Pentraxin, 020 7433 2786


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